Imetelstat Sodium in Treating Patients With Primary or Secondary Myelofibrosis
This pilot clinical trial studies how well imetelstat sodium works in treating patients with primary or secondary myelofibrosis. Imetelstat sodium may stop the growth of cancer cells by blocking some of the enzymes needed for cell growth
|Study Design:||Endpoint Classification: Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
|Official Title:||A Pilot Open-Label Study of the Efficacy and Safety of Imetelstat (GRN163L) in Patients With DIPSS_plus Intermediate-2 or High Risk Primary Myelofibrosis (PMF), Post-Polycythemia Vera Myelofibrosis (Post-PV MF), or Post-Essential Thrombocythemia Myelofibrosis (Post-ET-MF).|
- Overall response rate defined as a clinical improvement (CI), partial remission (PR), or complete remission (CR) according to the IWG-MRT consensus criteria [ Time Frame: Up to 27 weeks ] [ Designated as safety issue: No ]Will be estimated by the number of successes divided by the total number of evaluable patients. Ninety-five percent Duffy Santner confidence intervals for the true success proportion will be calculated.
- Maximum grade for each type of adverse event for each patient [ Time Frame: Up to 5 years ] [ Designated as safety issue: Yes ]Frequency tables will be reviewed to determine patterns. Additionally, the relationship of the adverse event(s) to the study treatment will be taken into consideration.
- Spleen response defined as either a minimum 50% reduction in palpable splenomegaly of a spleen that is at least 10 cm at baseline or a spleen that is palpable at more than 5 cm at baseline becomes not palpable [ Time Frame: Up to 27 weeks ] [ Designated as safety issue: No ]Will be calculated by the number of patients who achieve spleen response divided by the total number of evaluable patients with spleen involvement at baseline (estimated to be approximately 80% of patients). Ninety-five percent exact binomial confidence intervals for the true proportion of patients with spleen response will be calculated.
- Proportion of patients achieving transfusion independence [ Time Frame: Up to 27 weeks ] [ Designated as safety issue: No ]Will be estimated by the number of patients who achieve transfusion independence divided by the total number of evaluable patients who were transfusion dependent at baseline Ninety-five percent exact binomial confidence intervals for the true proportion of patients who become transfusion independent will be calculated.
|Study Start Date:||October 2012|
|Estimated Primary Completion Date:||March 2014 (Final data collection date for primary outcome measure)|
Experimental: Treatment (imetelstat sodium)
Patients receive imetelstat sodium IV over 2 hours on day 1. Treatment repeats every 21 days for up to 52 courses in the absence of disease progression or unacceptable toxicity.
Drug: imetelstat sodium
I. To evaluate overall response rate.
I. To evaluate the safety and tolerability of imetelstat (imetelstat sodium) in myelofibrosis (MF) (per common terminology criteria for adverse events, version 4.03).
II. To evaluate the efficacy of imetelstat in the reduction of spleen size, as measured by physical examination (palpable distance from the left costal margin).
III. To evaluate the efficacy of imetelstat in inducing red blood cell transfusion-independence in previously transfusion-dependent patients (per International Working Group for Myelofibrosis Research and Treatment [IWG-MRT] criteria).
I. To evaluate the effect of imetelstat on bone marrow histology and karyotype. II. To evaluate the effect of imetelstat on leukocytosis and thrombocytosis.
OUTLINE: Patients receive imetelstat sodium intravenously (IV) over 2 hours on day 1. Treatment repeats every 21 days for up to 52 courses in the absence of disease progression or unacceptable toxicity.
After completion of study treatment, patients are followed up every 6 months for 5 years.
|United States, Minnesota|
|Rochester, Minnesota, United States, 55905|
|Contact: Mayo Clinic Clinical Trials Referral Office 507-538-7623|
|Principal Investigator: Ayalew Tefferi, M.D.|
|Study Chair:||Ayalew Tefferi, M.D.||Mayo Clinic|